“Youth is wasted on the young.” That quote, often attributed to playwright George Bernard Shaw among others, may on the surface appear paradoxical. However, to me its meaning is clear. When we are young and in our physical prime, both the slate of options for physical…

I’ve been experimenting with improving my leg strength and gaining back a lost skill. I share the surprising results below. Since my ALS diagnosis in 2010, I’ve followed a daily practice of range-of-motion exercises based on fundamental movements taught by the strength and movement specialist company Original Strength. I also…

Stem cell therapy using a certain type of stem cell called human mesenchymal stromal cells (hMSCs) may have the potential to improve outcomes and prevent progression of familial amyotrophic lateral sclerosis (ALS), a mouse study suggests. Repeated administrations of these stem cells in the spinal cord prolonged the lifespan…

With each new advance in medicine comes ethical dilemmas, from fertility treatments and newborn screening, to vaccinations, gene therapies and euthanasia. But rare diseases and the expensive therapies needed to treat them — particularly in an age of scarce economic resources — almost always entail “tragic choices,” warned Avraham Steinberg,…

“She put me through some changes, Lord, sort of like a Waring blender. Poor, poor, pitiful me. Poor, poor, pitiful me.” –Warren Zevon *** The other day while I was ruminating about the burdens that ALS imposes, a call of nature provided an intervention of…

Rare diseases affect about 30 million Americans — roughly the same number as those with type 2 diabetes. Yet only 5 percent of the estimated 7,000 rare diseases known to science have cures or treatments approved by the U.S. Food and Drug Administration (FDA). Raising awareness of those illnesses and highlighting…

A modified experimental version of the TDP-43 protein has revealed the underlying mechanisms that support its accumulation and the formation of toxic aggregates implicated in amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD), a study reports. A detailed understanding of TDP-43-mediated neurotoxicity may help researchers find new targeted…

They’re boring, slow, and only for old people! That’s what I told my husband when he first suggested that I consider using a mobility scooter. Like so many who live with ALS symptoms, weak muscles in my lower legs limit my mobility. At home, I rely…

The world’s biggest gathering of rare disease researchers, patient groups, pharmaceutical executives, and government officials is planned for April 10–12 in a Washington, D.C., suburb. Some 1,200 people have already registered to attend the World Orphan Drug Congress (WODC) USA 2019, set to take place at the Gaylord National Harbor…

About 100 scientists, researchers, pharmaceutical executives, and others will converge on Austria’s capital city early next month for the 2nd International Congress on Advanced Treatments in Rare Diseases. The March 4-5 meeting, to take place at the Hilton Am Stadtpark Vienna, features 27 speakers on a variety of disorders…